US12569573B2ActiveUtilityA1

Enhanced transduction of AAV vectors encoding micrornas

56
Assignee: UNIV FLORIDAPriority: Apr 13, 2019Filed: Apr 13, 2020Granted: Mar 10, 2026
Est. expiryApr 13, 2039(~12.7 yrs left)· nominal 20-yr term from priority
Inventors:SRIVASTAVA ARUNQING KEYUN
C12N 2800/40C12N 2750/14142C12N 2750/14123C12N 2750/14122C12N 2310/141C12N 15/86C12N 15/113A61K 45/06C12N 2310/51C12N 2320/32A61K 48/0066C12N 2750/14143A61P 9/10A61P 9/00A61K 48/005
56
PatentIndex Score
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Cited by
33
References
11
Claims

Abstract

Provided herein are recombinant adeno-associated virus (rAAV) particles encoding microRNAs targeting the glucocorticoid receptor (GR) pathway, and in particular a microRNA17-92 (miR 17-92) cluster, and genes of interest. The modified genomes of these rAAV particles comprise heterologous nucleic acid sequences encoding microRNA structures. These particles exhibit enhanced transduction efficiencies in mammalian cells. Also provided herein are compositions of nucleic acids encoding the miR 17-92 cluster and nucleic acids encoding a gene of interest. Further provided herein are methods for administering these nucleic acid compositions to enhance transduction efficiencies.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A composition comprising:
 a) a first rAAV nucleic acid comprising a heterologous nucleic acid encoding two or more miRNA sequences that activate glucocorticoid receptor (GR) activity, wherein the two or more miRNA sequences are selected from SEQ ID NOs: 1-6 and 13-16; and   b) a second rAAV nucleic acid comprising a heterologous nucleic acid encoding a transgene of interest.   
     
     
         2 . The composition of  claim 1 , wherein the first rAAV nucleic acid and the second rAAV nucleic acid are single-stranded. 
     
     
         3 . The composition of  claim 1 , wherein the two or more miRNA sequences comprise a first sequence comprising SEQ ID NO: 1 and a second sequence comprising SEQ ID NO: 2. 
     
     
         4 . The composition of  claim 1 , wherein the first rAAV nucleic acid is encapsidated within a first rAAV particle and the second rAAV nucleic acid is encapsidated within a second rAAV particle. 
     
     
         5 . The composition of  claim 1  further comprising a tyrphostin. 
     
     
         6 . A method comprising contacting the composition of  claim 1  with a cell. 
     
     
         7 . The method of  claim 6 , wherein the cell is a mammalian cell. 
     
     
         8 . The method of  claim 7 , wherein the mammalian cell is a human cell. 
     
     
         9 . A method comprising administering the composition of  claim 1  to a subject. 
     
     
         10 . The method of  claim 9 , wherein the subject is a mammalian subject. 
     
     
         11 . The method of  claim 10 , wherein the mammalian subject is a human.

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